The F.D.A.’s New Boss Will Face an Age-Old Dilemma

In recent years, decisions made by the Food and Drug Administration have increasingly moved into the public eye. The agency oversees some of the most politically fraught treatments — abortion medication, opioids, Covid therapies and vaccines. Patients and caregivers are also more vocal about their desires, amplified by social media: You may have seen campaigns from families seeking F.D.A. approval for a drug that has a chance of treating their child’s rare disease.

Dr. Marty Makary, President Trump’s nominee to lead the F.D.A., would step into a role increasingly defined by the tension between fostering lifesaving innovation and ensuring that the public is protected from unsafe or ineffective drugs. This will be made even more complicated by the Trump administration’s threats to research funding and the distrust of science espoused by Dr. Makary’s boss, Robert F. Kennedy Jr.

The F.D.A. has long been maligned for being too slow in approving drugsfor patients with fatal diseases. But when the agency approves a treatment with less robust data to prove its effectiveness, it is maligned for being influenced by the pharmaceutical industry or patient advocacy groups. The rise of gene therapies and other targeted treatments that promise to shift the course of rare diseases will only make this balancing act harder: These therapies can’t necessarily be tested in traditional rigorous, large-scale trials because of the inherently small number of people with the diagnosis.

Patients have long pushed the F.D.A. on its decisions. In the late 1980s, as the AIDS crisis devastated America, activists surrounded the F.D.A. headquarters to protest what they perceived as overly slow and even obstructionist drug approval processes. Patients were dying as the F.D.A. was waiting for better data to decide whether to approve a drug. Activists argued that there had to be a way to get promising treatments into the hands of patients, outside of clinical trials, before studies were complete.

This pressure led the F.D.A. in the early 1990s to develop what’s called the accelerated approval pathway, which allows drugs for serious, incurable diseases with promising data to be granted approval while larger-scale studies continue. The approval is conditional, meaning that if the treatment doesn’t end up showing a benefit in follow-up studies, the drug can be taken off the market. This was a major step forward for patients, granting them access to drugs that might not have been available within their lifetimes.

But this, too, is an imperfect process. Studies have questioned how many of these drugs ultimately show success. For instance, a recent review of cancer drugs that were granted accelerated approval revealed that fewer than half of them helped patients in follow-up trials. And those that didn’t were slow to be removed from the market.